Cora is a very energetic new starter for Prime Global, so it was great to pick her brains and get to know more about her career path. Cora has so much experience and knowledge in the industry, we are excited that she is part of our team to drive Onyx forward!

Meet Tim. Tim is Tom’s little brother. From birth he had some health complications, but quickly recovered. However, like Tom he had trouble with his speech at the age of 2. The explanation? “He’s just like Tom.” Read more about brothers Tim and Tom on our blog and follow the patient journey and lengthy diagnosis for a child living with a rare disease.

Duchenne muscular dystrophy is a devastating disease that we still don’t fully understand, and currently there is no cure. This article describes current and new treatment therapies as well as the many challenges scientists still face. https://lnkd.in/gYkEcuH

Cystic fibrosis is a rare disease that causes damage to the respiratory, digestive and reproductive systems. Our recent article presents what is currently known about this disease as well as existing and future therapies. https://lnkd.in/gkMERE4

Tom and Tim finally reach a diagnosis after finding genetic faults known as AGU. The doctor couldn’t tell them much more, so they were sent to a rare disease specialist for further consultations. For Tom and Tim, and their families, it’s been a long process to reach a clear diagnosis. Sadly, like many other rare diseases, there is no therapy or treatment available. Rare diseases affect more than 350 million people worldwide. We need to support people like Tom and Tim to raise awareness of the long and stressful journey through to diagnosis, to understand the reality of living with a rare condition and to support those families affected by a rare disease.

This week we meet Tilly, Tom and Tim’s younger sister. She was born with no complications and breezed through her early years with good health. As she starts to grow though, you can see that Tilly looks different to her brothers. Read more about their journey so far on our blog: https://lnkd.in/dzd4_T8

Rare Disease Patients’ Journey: As Tom and Tim grow, more symptoms arise. Tom is struggling to keep up in class and not progressing academically at the same rate as his class mates. It’s also being noticed that Tim is struggling with his hearing. Read more about their journey so far on our blog: https://lnkd.in/dzd4_T8

A person living with a rare disease will have a full diagnosis of an average of 4.8 years. We’re following the journey of brothers Tim and Tom, who are at the beginning of their symptoms. Their story, as well as hundreds and thousands like them #NeedToBeHeard.

We want to share what it’s like to live with a #RareDisease and show how long it can take for a patient to get diagnosed. Follow our blog:https://lnkd.in/dzd4_T8 to read about Tom and how his symptoms develop over time. Our first post shares his parents’ concern about the initial symptoms in the first two years of his life. #NotSoRare

Today is #RareDiseaseDay. Did you know a patient will end up visiting several doctors before getting an accurate diagnosis? Let’s work together to heighten awareness #notsoRARE Engel PA, Bagal S, Broback M, Boice N. Physician and patient perceptions regarding physician training in rare diseases: The need for stronger educational initiatives for physicians. Journal of Rare Disorders, 2013; 1(2): 1-15.

In support of #RareDiseaseDay we are launching a series of blog posts to illustrate the length of time it takes for a child living with a rare disease to receive a diagnosis. The process will be reviewed, from the parent’s perspective looking at the initial symptoms and beyond. Follow the link to find out about Tom. https://onyx-notsorare.tumblr.com/ Stay tuned over the next couple of weeks to find out what happens. #notsoRARE Engel PA, Bagal S, Broback M, Boice N. Physician and patient perceptions regarding physician training in rare diseases: The need for stronger educational initiatives for physicians.

Prime GlobalAmazing scientific sight…! The teams from ONYX and Only Oncology take a few moments on a recent business trip to admire the Atomium as it gleams against the bright blue Brussels sky!02.06.2017

The first drug to treat the underlying cause of Duchenne muscular dystrophy, Exondys (eteplirsen) developed by Sarepta Therapeutics, has secured marketing approval from the U.S. Food and Drug Administration. Duchenne muscular dystrophy is a severe type of muscular dystrophy which generally begins around age four in boys and progresses rapidly.

Strimvelis, (autologous CD34+ cells transduced to express ADA) the first ex vivo stem cell gene therapy has been approved to treat patients with ADA-SCID (severe combined immunodeficiency due to adenosine deaminase deficiency). Children born with ADA-SCID do not have a heathy, fully functioning immune system and are unable to successfully combat everyday infections.

Following a successful presentation at the Cell Therapy Manufacturing and Gene Therapy Congress in Brussels, Onyx has again had a strong presence at the prestigious World Orphan Drugs Congress in Washington. This emphasises Onyx strong and continuing commitment to the areas of orphan drugs, rare and ultra-rare diseases and regenerative medicine including cell and gene therapies.